Journal
ANNUAL REVIEW OF BIOMEDICAL ENGINEERING, VOL 13
Volume 13, Issue -, Pages 507-530Publisher
ANNUAL REVIEWS
DOI: 10.1146/annurev-bioeng-071910-124709
Keywords
RNA interference; siRNA; cationic lipid; gene therapy
Categories
Funding
- NATIONAL CANCER INSTITUTE [R01CA129835, R01CA129421, R01CA149363] Funding Source: NIH RePORTER
- NCI NIH HHS [CA149363, CA129835, CA129421] Funding Source: Medline
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RNA interference (RNAi) technology represents a fundamentally new category of treatments for human disease by addressing targets that are traditionally considered undruggable with existing medicines. The major challenge for RNAi-based therapy is the delivery system that meets human therapeutic needs. Therefore, engineering vectors with good delivery efficiency and safety profile is an intense area of research. Lipid-based nanoparticles for RNAi have yielded successful advances in vivo and to an extent in clinical trials. In this review, we discuss the barriers in developing lipid-based nanoparticles for in vivo RNAi and different strategies to overcome them. Rational designs that address safety concerns and ensure effective delivery will aid the translation of engineered lipid-based nanoparticles toward the clinic in the foreseeable future.
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